A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term

• Register: EUCTR
• Last refreshed on: 20 August 2018
• Main ID: EUCTR2018-000156-18-ES
• Date of registration: 14/05/2018
• Prospective Registration: Yes
• Primary sponsor: Ultragenyx Pharmaceutical, Inc.
• Public title: A clinical study to learn about the effects of a virus that transfers the gene for human Ornithine Transcarbamylase (OTC) in adults with late-onset OTC deficiency in the long term
• Scientific title: A Long-Term Follow-up Study to Evaluate the Safety and Efficacy of Adeno-Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Adults with Late-Onset OTC Deficiency
• Date of first enrolment: 18/06/2018
• Target sample size: 9
• Recruitment status: Authorised-recruitment may be ongoing or finished
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-000156-18
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): no Therapeutic use (Phase IV): no

Countries of recruitment

Canada, Spain, United Kingdom, United States

Contacts

• Name: Clinical Development
• Affiliation: Ultragenyx Pharmaceutical, Inc.

• Name: Clinical Development
• Affiliation: Ultragenyx Pharmaceutical, Inc.

Key inclusion & exclusion criteria

Inclusion criteria:
1. Completed the Week 52 visit in Study 301OTC01. Note that the Day 0 visit of Study 301OTC02 may coincide with the Week 52 visit of Study 301OTC01.
2. Willing and able to provide written informed consent.
3. Willing, able, and committed to comply with scheduled study site visits, study procedures, and requirements.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 8
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1

Exclusion criteria:
1. Planned or current participation in another interventional clinical study that may confound the efficacy or safety evaluation of DTX301 during the duration of this study.
2. Any clinically significant medical condition that, in the opinion of the investigator, would pose a risk to subject safety or would impede the study.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Ornithine transcarbamylase deficiency
MedDRA version: 20.0 Level: LLT Classification code 10071107 Term: Ornithine transcarbamylase deficiency System Organ Class: 100000004850

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Intervention(s)

Product Name: DTX301
Pharmaceutical Form: Concentrate for solution for infusion
INN or Proposed INN: Not yet assigned
Other descriptive name: DTX301
Concentration unit: Other
Concentration type: not less then
Concentration number: 5000000000000-

Primary Outcome(s)

Main Objective: To determine the long-term safety of DTX301 following a single IV dose in adults with late-onset OTC deficiency.

Timepoint(s) of evaluation of this end point: From the time the subject signs the Informed Consent Form through the end of study/early withdrawal visit.

Primary end point(s): The incidence of AEs and SAEs for each dosing cohort assessed by severity and relationship to study product.

Secondary Objective: To evaluate the long-term efficacy of DTX301 on AUC0-24 for plasma ammonia following a single IV dose in adults with late-onset OTC deficiency.

To evaluate the long-term effects of DTX301 on the rate of ureagenesis in adults with late-onset OTC deficiency.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Plasma ammonia: Day 0, Week 52, Week 104, Week 156, Week 208

Ureagenesis: Day 0, Week 26, Week 52, Week 78, Week 104, Week 130, Week 156, Week 182, Week 208

Secondary end point(s): The change from baseline (Day 0 of Study 301OTC01) in AUC0-24 for plasma ammonia over time to 260 weeks following IV administration of DTX301.

The change from baseline (average of Screening and Day 1 results of Study 301OTC01) in the rate of ureagenesis (as measured by the generation of [13C]urea over 4 hours) as determined by gas chromatography mass spectrometry over time to 260 weeks following IV administration of DTX301.

Secondary ID(s)

17190

301OTC02

Source(s) of Monetary Support

Ultragenyx Pharmaceutical, Inc.